NATIONAL MARROW DONOR PROGRAM

ACCESS Trial Shows Promising Survival Rates with PTCy GVHD Prophylaxis in HLA-Mismatched Transplants

3 months ago

• Phase 2 ACCESS trial demonstrates 84% one-year overall survival rate using post-transplant cyclophosphamide for GVHD prevention in HLA-mismatched unrelated donor transplants. • Study shows improved GVHD-free, relapse-free survival rate of 48% compared to 38% in previous 15-UUMD trial, marking significant progress in transplant outcomes. • Results indicate successful outcomes regardless of number of HLA mismatches, potentially expanding treatment options for patients requiring stem cell transplantation.

Enhertu Approved for HER2-Low and HER2-Ultralow Metastatic Breast Cancer

5 months ago

• The FDA has approved Enhertu for HR-positive, HER2-low or HER2-ultralow metastatic breast cancer after endocrine therapy progression. • DESTINY-Breast06 trial data showed a 36% reduction in disease progression or death risk compared to chemotherapy. • Patients on Enhertu had a median progression-free survival of 13.2 months versus 8.1 months on chemotherapy. • This approval expands Enhertu's use to an earlier treatment setting and a broader patient population.

AbbVie's Tavapadon Shows Positive Results in Phase 3 Parkinson's Disease Trial

6 months ago

• AbbVie's tavapadon met its primary endpoint in the TEMPO-2 Phase 3 trial, demonstrating significant improvement in Parkinson's disease symptoms and quality of life. • Patients treated with flexible doses of tavapadon (5-15mg daily) showed a 10.3-point improvement on the MDS-UPDRS scale compared to a 1.2-point worsening in the placebo group. • Tavapadon, which selectively activates D1 and D5 dopamine receptors, is planned for FDA submission next year, marking a potential new treatment option for Parkinson's. • This positive outcome contrasts with recent failures in AbbVie's schizophrenia drug program, highlighting the importance of tavapadon in their pipeline.

CAST Regimen Improves Outcomes in Half-Matched Stem Cell Transplants

7 months ago

• A new CAST regimen, combining post-transplant cyclophosphamide, abatacept, and tacrolimus, shows promise in preventing graft-versus-host disease (GVHD) after haploidentical hematopoietic stem cell transplantation (HSCT). • The CAST regimen demonstrated a statistically significant reduction in relapse rates and improved disease-free survival compared to the standard GVHD prevention regimen. • The study suggests CAST can improve outcomes in haploidentical HSCT, potentially reducing disparities for patients lacking readily available matched donors. • Researchers advocate for further investigation of the CAST regimen in a randomized controlled trial to confirm its benefits.

Real-World Study Affirms Axicabtagene Ciloleucel's Efficacy in LBCL, Expanding Patient Eligibility

3 years ago

• A real-world study of 1297 patients confirms the efficacy of axicabtagene ciloleucel (axi-cel) in relapsed/refractory large B-cell lymphoma (LBCL). • Patients ineligible for the ZUMA-1 trial, often due to comorbidities, still experienced durable responses with axi-cel treatment. • Elderly patients (≥65 years) showed favorable overall response rates despite a higher incidence of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). • The study suggests that patient selection for axi-cel should prioritize risk-to-benefit ratio and comorbidities over strict adherence to ZUMA-1 eligibility criteria.